E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10019860 |
E.1.2 | Term | Hereditary angioedema |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
•To assess the safety and tolerability of ecallantide in paediatric patients treated for acute attacks of HAE |
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E.2.2 | Secondary objectives of the trial |
•To evaluate the PK profile of ecallantide in paediatric patients treated for acute attacks of HAE •To assess the efficacy of ecallantide in paediatric patients treated for moderate (interferes with normal daily activities) to severe (inability to perform normal daily activities) acute attacks of HAE
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Each patient must meet the following criteria:
1.Male or female patient from 2 years of age and prepubertal (up through Tanner Stage 1; at the screening visit) 2.Physician diagnosis and history of HAE (Type I or II) including laboratory results showing C1-INH activity below the lower limit of normal or up to 15% above the lower limit of the normal range, as defined by the reference laboratory (at the screening visit) 3.Present at the site with moderate (interferes with normal daily activities) to severe (inability to perform normal daily activities) signs and symptoms of an acute attack of HAE within 8 hours of recognition of the onset of the attack (at pre-dose of Parts 1, 2, and 3). Spontaneous resolution of the attack must not have begun before the administration of study drug 4.Must have a signed informed consent by parent or caregiver
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E.4 | Principal exclusion criteria |
Patients who meet any of the following criteria will be excluded from the study. 1.Are <2 years of age or have reached puberty (as assessed by Tanner Stage 2; at the screening visit) 2.Received treatment with ecallantide within previous 72 hours (at pre-dose of Parts 1, 2, and 3) 3.Received an investigational (non-licensed) drug or device, other than ecallantide, within 30 days prior to the screening visit 4.For Part 2 only: pharyngeal/laryngeal symptoms (at pre-dose of Part 2) 5.Mild attacks including mild edema of the extremities and mild abdominal attacks (at pre-dose of Parts 1, 2, and 3) 6.Are unable or unwilling to give informed consent (patient or caregiver) 7.Any other condition that, in the opinion of the investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study (at the screening visit and at pre-dose of Parts 1, 2, and 3)
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety and tolerability of ecallantide. This will include vital signs, electrocardiogram (ECG), physical examination, clinical laboratory parameters (serum chemistry including liver function tests, haematology, coagulation, anti ecallantide antibodies), and adverse events (AEs). |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 9 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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As stated in the Protocol |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |